A dose of 3.5 million dollars .. FDA approves the use of the “most expensive drug in the world”

24 November, 2022
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A dose of 3.5 million dollars .. FDA approves the use of the “most expensive drug in the world”

The US Food and Drug Administration (FDA) has approved the marketing of the world's most expensive drug for the treatment of the genetic disorder - Haemophilia B.

And so, the agency gave the green light to Hemgenix from biopharmaceutical company CSL Behring, which set its price at $3.5 million per dose, making it the most expensive treatment in the world. Hemgenix is ​​given intravenously, once, to adult patients with hemophilia B, an inherited blood disorder that affects mostly men and is caused by a genetic mutation in a protein essential for blood clotting. If left untreated, the condition can cause bleeding that leaks into organs and joints, including the brain. A study found that it cut the number of expected bleeding episodes over the course of a year by more than half, and freed 94% of patients from the need for regular injections to keep things under control. 

"Gene therapy for hemophilia has been on the horizon for more than two decades," said Peter Marks, director of the FDA's Center for Biology Research Evaluation, noting that the approval "represents an important advance in developing innovative therapies for those with a high burden of the disease." associated with this form of hemophilia.

Hemophilia B accounts for about 15% of patients with hemophilia, and affects approximately one in every 40,000 population. Women with the disease often do not show any symptoms.

Hemgenix consists of a viral vector that carries the gene for coagulation factor IX (factor IX), which is expressed in the liver in order to increase levels of this enzyme in the blood and thus reduce bleeding episodes. Given that this new gene therapy is so expensive, its cost has raised questions about whether it will ever be widely adopted.

Brad Loncar, a biotechnology investor and CEO of Loncar Investments, told Bloomberg: “While the price came in a little higher than expected, I think it has a chance of success. First, because current drugs are also very expensive. And secondly, because patients with hemophilia always live in a cycle of fear of bleeding.” The US Food and Drug Administration said Hemgenix "represents an important advance in developing innovative therapies for people affected by the disease" by delivering a gene that can produce missing blood clotting factors in the liver. Conventional treatments pump in missing proteins, or clotting factors, that the body needs to form clots and stop bleeding.

It is estimated that about 16 million people in the United States and Europe have hemophilia B, while hemophilia A, the most common, affects five times that number.


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